DK1414996T3 - Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells - Google Patents
Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified CellsInfo
- Publication number
- DK1414996T3 DK1414996T3 DK02746963.4T DK02746963T DK1414996T3 DK 1414996 T3 DK1414996 T3 DK 1414996T3 DK 02746963 T DK02746963 T DK 02746963T DK 1414996 T3 DK1414996 T3 DK 1414996T3
- Authority
- DK
- Denmark
- Prior art keywords
- cells
- methods
- hematopoietic progenitor
- genetically modifying
- modified
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
- C12N15/1132—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/18—Antivirals for RNA viruses for HIV
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/121—Hammerhead
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- AIDS & HIV (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Tropical Medicine & Parasitology (AREA)
- Epidemiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US30412701P | 2001-07-10 | 2001-07-10 | |
US30428301P | 2001-07-10 | 2001-07-10 | |
US34348401P | 2001-12-21 | 2001-12-21 | |
US38606302P | 2002-06-04 | 2002-06-04 | |
PCT/US2002/021907 WO2003006691A1 (en) | 2001-07-10 | 2002-07-10 | Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells |
Publications (1)
Publication Number | Publication Date |
---|---|
DK1414996T3 true DK1414996T3 (en) | 2011-03-21 |
Family
ID=27501852
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK02746963.4T DK1414996T3 (en) | 2001-07-10 | 2002-07-10 | Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells |
Country Status (16)
Country | Link |
---|---|
US (2) | US7345025B2 (en) |
EP (2) | EP2322618A1 (en) |
JP (2) | JP5179697B2 (en) |
KR (1) | KR101015913B1 (en) |
CN (2) | CN1553963A (en) |
AT (1) | ATE490322T1 (en) |
AU (1) | AU2002316640C1 (en) |
BR (1) | BR0211079A (en) |
CA (1) | CA2453531A1 (en) |
DE (1) | DE60238486D1 (en) |
DK (1) | DK1414996T3 (en) |
HK (1) | HK1065074A1 (en) |
IL (2) | IL159637A0 (en) |
MX (1) | MXPA04000268A (en) |
NZ (1) | NZ530624A (en) |
WO (1) | WO2003006691A1 (en) |
Families Citing this family (40)
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US20020058636A1 (en) * | 1994-09-21 | 2002-05-16 | Geoffrey P. Symonds | Ribozymes targeting the retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs |
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US6627442B1 (en) * | 2000-08-31 | 2003-09-30 | Virxsys Corporation | Methods for stable transduction of cells with hiv-derived viral vectors |
IL159777A0 (en) * | 2001-07-10 | 2004-06-20 | J & J Res Pty Ltd | Production of transduced hematopoietic progenitor cells |
ES2358187T3 (en) | 2001-07-10 | 2011-05-06 | JOHNSON & JOHNSON RESEARCH PTY LIMITED | PROCEDURES FOR THE GENETIC MODIFICATION OF HEMATOPOYETIC PROGENITING CELLS AND USES OF THE MODIFIED CELLS. |
CA2453531A1 (en) | 2001-07-10 | 2003-01-23 | Johnson & Johnson Research Pty Limited | Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells |
US7737124B2 (en) | 2001-09-13 | 2010-06-15 | California Institute Of Technology | Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell |
DE60233333D1 (en) * | 2001-09-13 | 2009-09-24 | California Inst Of Techn | METHOD FOR EXPRESSING SMALL ANTIVIRAL RNA MOLECULES WITHIN ONE CELL |
DE10212892A1 (en) * | 2002-03-20 | 2003-10-09 | Basf Plant Science Gmbh | Constructs and methods for regulating gene expression |
DE10224750A1 (en) | 2002-06-04 | 2003-12-24 | Fresenius Medical Care De Gmbh | Device for the treatment of a medical fluid |
WO2004071418A2 (en) * | 2003-02-06 | 2004-08-26 | Walter Reed Army Institute Of Research | Compounds for the treatment of systemic lupus erythematosus |
EP1613742A2 (en) * | 2003-04-08 | 2006-01-11 | YEDA RESEARCH AND DEVELOPMENT Co. LTD. | Stem cells having increased sensitivity to a chemoattractant and methods of generating and using same |
GB0410130D0 (en) * | 2004-05-06 | 2004-06-09 | Molmed Spa | Cell preparation |
US7935074B2 (en) * | 2005-02-28 | 2011-05-03 | Fresenius Medical Care Holdings, Inc. | Cassette system for peritoneal dialysis machine |
US8197231B2 (en) | 2005-07-13 | 2012-06-12 | Purity Solutions Llc | Diaphragm pump and related methods |
WO2007011088A1 (en) * | 2005-07-20 | 2007-01-25 | Seoul National University Industry Foundation | Method for culturingand proliferating hematopoietic stem cells and progenitor cells using human endometrial cells |
EP2032691A2 (en) * | 2006-06-15 | 2009-03-11 | Neostem, Inc | Processing procedure for peripheral blood stem cells |
JP2011160661A (en) * | 2008-06-02 | 2011-08-25 | Kyowa Hakko Kirin Co Ltd | Reprogramming method of blood cell |
US8112365B2 (en) * | 2008-12-19 | 2012-02-07 | Foster Scott C | System and method for online employment recruiting and evaluation |
US20100210989A1 (en) | 2008-12-23 | 2010-08-19 | Janet Lesley Macpherson | Processing blood |
US8192401B2 (en) | 2009-03-20 | 2012-06-05 | Fresenius Medical Care Holdings, Inc. | Medical fluid pump systems and related components and methods |
WO2010117974A2 (en) * | 2009-04-09 | 2010-10-14 | Stemcyte Inc. | Hiv-resistant stem cells and uses thereof |
CA2767668C (en) | 2009-07-15 | 2017-03-07 | Fresenius Medical Care Holdings, Inc. | Medical fluid cassettes and related systems and methods |
US8720913B2 (en) | 2009-08-11 | 2014-05-13 | Fresenius Medical Care Holdings, Inc. | Portable peritoneal dialysis carts and related systems |
JP5946775B2 (en) * | 2010-12-01 | 2016-07-06 | 日産化学工業株式会社 | Method for producing hematopoietic stem cells using pyrazole compounds |
DE102010053973A1 (en) | 2010-12-09 | 2012-06-14 | Fresenius Medical Care Deutschland Gmbh | Medical device with a heater |
US9694125B2 (en) | 2010-12-20 | 2017-07-04 | Fresenius Medical Care Holdings, Inc. | Medical fluid cassettes and related systems and methods |
US9624915B2 (en) | 2011-03-09 | 2017-04-18 | Fresenius Medical Care Holdings, Inc. | Medical fluid delivery sets and related systems and methods |
JP6062920B2 (en) | 2011-04-21 | 2017-01-18 | フレセニウス メディカル ケア ホールディングス インコーポレーテッド | Medical fluid pumping system and related devices and methods |
US9186449B2 (en) | 2011-11-01 | 2015-11-17 | Fresenius Medical Care Holdings, Inc. | Dialysis machine support assemblies and related systems and methods |
US9610392B2 (en) | 2012-06-08 | 2017-04-04 | Fresenius Medical Care Holdings, Inc. | Medical fluid cassettes and related systems and methods |
US9500188B2 (en) | 2012-06-11 | 2016-11-22 | Fresenius Medical Care Holdings, Inc. | Medical fluid cassettes and related systems and methods |
CN113897398A (en) * | 2012-09-07 | 2022-01-07 | 再生元制药公司 | Genetically modified non-human animals and methods of use thereof |
DK3939423T3 (en) * | 2012-11-05 | 2024-05-06 | Regeneron Pharma | Genetically modified non-human animals and methods of use thereof |
JP6483023B2 (en) * | 2012-12-06 | 2019-03-13 | エンリヴェックス セラピューティクス リミテッド | A therapeutic apoptotic cell preparation, its production method and its use |
US9561323B2 (en) | 2013-03-14 | 2017-02-07 | Fresenius Medical Care Holdings, Inc. | Medical fluid cassette leak detection methods and devices |
US10117985B2 (en) | 2013-08-21 | 2018-11-06 | Fresenius Medical Care Holdings, Inc. | Determining a volume of medical fluid pumped into or out of a medical fluid cassette |
CN103471896A (en) * | 2013-09-09 | 2013-12-25 | 上海兰卫临床检验有限公司 | Preparation method for gram staining solution quality control product |
US20200199534A1 (en) * | 2017-04-27 | 2020-06-25 | Fred Hutchinson Cancer Research Center | Therapeutic formulations containing cd34+ stem cells derived from negative selection |
SG11202004145QA (en) * | 2017-11-15 | 2020-06-29 | Weird Science Llc | Methods and compositions for non-myeloablative bone marrow reconstitution |
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US5712384A (en) * | 1994-01-05 | 1998-01-27 | Gene Shears Pty Ltd. | Ribozymes targeting retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs |
EP0753062A4 (en) | 1994-01-05 | 1999-10-20 | Gene Shears Pty Ltd | Ribozymes targeting the retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs |
US20020058636A1 (en) | 1994-09-21 | 2002-05-16 | Geoffrey P. Symonds | Ribozymes targeting the retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs |
EP0821740A1 (en) | 1995-04-20 | 1998-02-04 | Chiron Corporation | High efficiency ex vivo transduction of hematopoietic stem cells by recombinant retroviral preparations |
US5741706A (en) * | 1996-06-13 | 1998-04-21 | Immusol, Incorporated | Anti-HIV ribozymes |
TWI239352B (en) * | 1997-07-23 | 2005-09-11 | Takara Bio Inc | Gene transfer method with the use of serum-free medium |
US6506559B1 (en) * | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
US6060317A (en) * | 1998-08-11 | 2000-05-09 | The United States Of America As Represented By The Department Of Health And Human Services | Method of transducing mammalian cells, and products related thereto |
WO2000034495A2 (en) * | 1998-12-04 | 2000-06-15 | City Of Hope | A method of genetically modifying very primitive quiescent human hematopoietic stem cells |
AU5403100A (en) * | 1999-06-08 | 2000-12-28 | Novartis Ag | Enforced expression of bcl-xl |
JP2004509120A (en) | 2000-09-13 | 2004-03-25 | バーテックス ファーマシューティカルズ インコーポレイテッド | Caspase inhibitors and their uses |
US20020160393A1 (en) | 2000-12-28 | 2002-10-31 | Symonds Geoffrey P. | Double-stranded RNA-mediated gene suppression |
IL159777A0 (en) | 2001-07-10 | 2004-06-20 | J & J Res Pty Ltd | Production of transduced hematopoietic progenitor cells |
CA2453531A1 (en) | 2001-07-10 | 2003-01-23 | Johnson & Johnson Research Pty Limited | Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells |
ES2358187T3 (en) | 2001-07-10 | 2011-05-06 | JOHNSON & JOHNSON RESEARCH PTY LIMITED | PROCEDURES FOR THE GENETIC MODIFICATION OF HEMATOPOYETIC PROGENITING CELLS AND USES OF THE MODIFIED CELLS. |
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-
2002
- 2002-07-10 CA CA002453531A patent/CA2453531A1/en not_active Abandoned
- 2002-07-10 IL IL15963702A patent/IL159637A0/en unknown
- 2002-07-10 NZ NZ530624A patent/NZ530624A/en not_active IP Right Cessation
- 2002-07-10 EP EP10183035A patent/EP2322618A1/en not_active Withdrawn
- 2002-07-10 MX MXPA04000268A patent/MXPA04000268A/en active IP Right Grant
- 2002-07-10 AU AU2002316640A patent/AU2002316640C1/en not_active Ceased
- 2002-07-10 BR BR0211079-2A patent/BR0211079A/en not_active IP Right Cessation
- 2002-07-10 KR KR1020047000421A patent/KR101015913B1/en not_active IP Right Cessation
- 2002-07-10 CN CNA028177428A patent/CN1553963A/en active Pending
- 2002-07-10 CN CN2010102736257A patent/CN101926824A/en active Pending
- 2002-07-10 EP EP02746963A patent/EP1414996B1/en not_active Expired - Lifetime
- 2002-07-10 JP JP2003512448A patent/JP5179697B2/en not_active Expired - Fee Related
- 2002-07-10 DK DK02746963.4T patent/DK1414996T3/en active
- 2002-07-10 DE DE60238486T patent/DE60238486D1/en not_active Expired - Lifetime
- 2002-07-10 AT AT02746963T patent/ATE490322T1/en not_active IP Right Cessation
- 2002-07-10 WO PCT/US2002/021907 patent/WO2003006691A1/en active Application Filing
- 2002-07-10 US US10/192,980 patent/US7345025B2/en not_active Expired - Fee Related
-
2003
- 2003-12-29 IL IL159637A patent/IL159637A/en not_active IP Right Cessation
-
2004
- 2004-10-14 HK HK04107955.4A patent/HK1065074A1/en not_active IP Right Cessation
-
2006
- 2006-08-18 US US11/506,722 patent/US7776595B2/en not_active Expired - Fee Related
-
2009
- 2009-09-14 JP JP2009211662A patent/JP2010077125A/en active Pending
Also Published As
Publication number | Publication date |
---|---|
HK1065074A1 (en) | 2005-02-08 |
IL159637A0 (en) | 2004-06-01 |
EP1414996B1 (en) | 2010-12-01 |
KR20040032852A (en) | 2004-04-17 |
JP2005528319A (en) | 2005-09-22 |
CN101926824A (en) | 2010-12-29 |
US7776595B2 (en) | 2010-08-17 |
US20080044394A1 (en) | 2008-02-21 |
BR0211079A (en) | 2005-08-30 |
KR101015913B1 (en) | 2011-02-23 |
NZ530624A (en) | 2009-02-28 |
US7345025B2 (en) | 2008-03-18 |
EP1414996A1 (en) | 2004-05-06 |
EP2322618A1 (en) | 2011-05-18 |
JP2010077125A (en) | 2010-04-08 |
DE60238486D1 (en) | 2011-01-13 |
ATE490322T1 (en) | 2010-12-15 |
CN1553963A (en) | 2004-12-08 |
WO2003006691A1 (en) | 2003-01-23 |
AU2002316640C1 (en) | 2009-01-29 |
MXPA04000268A (en) | 2005-03-07 |
IL159637A (en) | 2013-07-31 |
EP1414996A4 (en) | 2007-03-28 |
CA2453531A1 (en) | 2003-01-23 |
JP5179697B2 (en) | 2013-04-10 |
US20040072771A1 (en) | 2004-04-15 |
AU2002316640B2 (en) | 2008-06-12 |
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